Hypercholesterolemia, FamilialPhase 2NCT00281008
Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
A Phase 2 study of Hypercholesterolemia, Familial, run by Kastle Therapeutics, LLC at 8 sites in 2 countries.
Trial summary
The aim of this study is to assess the safety and efficacy of varying doses of ISIS 301012 (mipomersen) as add-on therapy in subjects with Heterozygous Familial Hypercholesterolemia Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hypercholesterolemia, Familial
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 8 sitesin 2 countries
- Study dates
- Feb 2006to Dec 2007
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Weight ≥ 50 kg
- Diagnosis of Heterozygous Familial Hypercholesterolemia.
- Females must be non-pregnant and non-lactating.
- On stable lipid lowering therapy for at least 4 weeks.
- Lipid levels meet the pre-specified criteria.
Who cannot join
- Subject had heart problems in the prior 6 months.
- Subject has elevated ALT, AST, or CPK.
- History of renal disease, liver disease, or malignancy.
- Use of oral anticoagulants, unless the dose has been stable for 4 weeks
- Have any other conditions, which in the opinion of the Investigator would make the subject unsuitable for enrollment, or could interfere with the subject participating in or completing the study.
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
United States7 sites
Study site
Auburn, Maine, 04210
Study site
Chicago, Illinois, 60610
Study site
Cincinnati, Ohio, 45229
Study site
Houston, Texas, 77030
Study site
New York, New York, 10021
Study site
Scarborough, Maine, 04074
Study site
Winston-Salem, North Carolina, 27103
About this study
Official title: A Phase 2, Double-Blind, Placebo-Controlled, Dose Escalation Study to Assess the Safety, Efficacy, and Pharmacokinetics of ISIS 301012 as Add-on Therapy in Heterozygous Familial Hypercholesterolemia Subjects
The aim of this study is to assess the safety and efficacy of varying doses of ISIS 301012 (mipomersen) as add-on therapy in subjects with Heterozygous Familial Hypercholesterolemia
- Conditions
- Hypercholesterolemia, Familial
- Being tested
- ISIS 301012 or Placebo
- Keywords
- LDL-cholesterol, apoB-100, Heterozygous Familial Hypercholesterolemia
- Medical subject headings
- Hyperlipoproteinemia Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ISIS 301012 or PlaceboDrug
50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
ISIS 301012 or PlaceboDrug
100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
ISIS 301012 or PlaceboDrug
200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
ISIS 301012 or PlaceboDrug
300 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, 36, 43, 50, 57, 64, 71, 78, and 85
| Study group | Role | What they receive |
|---|---|---|
| Cohort A | Experimental | Loading doses followed by weekly maintenance doses Receives: Drug: ISIS 301012 or Placebo |
| Cohort B | Experimental | Loading doses followed by weekly maintenance doses Receives: Drug: ISIS 301012 or Placebo |
| Cohort C | Experimental | Loading doses followed by weekly maintenance doses Receives: Drug: ISIS 301012 or Placebo |
| Cohort D | Experimental | Loading doses followed by extended weekly maintenance doses Receives: Drug: ISIS 301012 or Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent reduction in LDL-cholesterol from baseline
Measured: Week 7 (Cohorts A-C), Week 15 (Cohort D)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2006 (estimated)
- Study startFeb 2006
- Primary completionJul 2007
- Study completionDec 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypercholesterolemia, FamilialStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kastle Therapeutics, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ionis Pharmaceuticals, Inc.
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00281008
- Sponsor's protocol ID
- 301012CS9
- Other IDs
- 2005-004797-24, 2005
- Status last verified
- Aug 2016
- First submitted
- 20 Jan 2006
- Last updated
- 3 Aug 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Double-Blind, Placebo-Controlled, Dose Escalation Study to Assess the Safety, Efficacy, and Pharmacokinetics of ISIS 301012 as Add-on Therapy in Heterozygous Familial Hypercholesterolemia Subjects
- ClinicalTrials.gov ID
- NCT00281008
- Acronym / sponsor ID
- 301012CS9
- Conditions
- Hypercholesterolemia, Familial
- Keywords
- LDL-cholesterol, apoB-100, Heterozygous Familial Hypercholesterolemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Kastle Therapeutics, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Ionis Pharmaceuticals, Inc.
- Study start
- Feb 2006
- Primary completion
- Jul 2007 (actual)
- Study completion
- Dec 2007 (actual)
- First posted
- 24 Jan 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to assess the safety and efficacy of varying doses of ISIS 301012 (mipomersen) as add-on therapy in subjects with Heterozygous Familial Hypercholesterolemia
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort A | Experimental | Drug: ISIS 301012 or PlaceboLoading doses followed by weekly maintenance doses |
| Cohort B | Experimental | Drug: ISIS 301012 or PlaceboLoading doses followed by weekly maintenance doses |
| Cohort C | Experimental | Drug: ISIS 301012 or PlaceboLoading doses followed by weekly maintenance doses |
| Cohort D | Experimental | Drug: ISIS 301012 or PlaceboLoading doses followed by extended weekly maintenance doses |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Weight ≥ 50 kg
- Diagnosis of Heterozygous Familial Hypercholesterolemia.
- Females must be non-pregnant and non-lactating.
- On stable lipid lowering therapy for at least 4 weeks.
- Lipid levels meet the pre-specified criteria.
Key exclusion criteria (5 listed)
- Subject had heart problems in the prior 6 months.
- Subject has elevated ALT, AST, or CPK.
- History of renal disease, liver disease, or malignancy.
- Use of oral anticoagulants, unless the dose has been stable for 4 weeks
- Have any other conditions, which in the opinion of the Investigator would make the subject unsuitable for enrollment, or could interfere with the subject participating in or completing the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ISIS 301012 or Placebo50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36 | Drug | Cohort A |
| ISIS 301012 or Placebo100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36 | Drug | Cohort B |
| ISIS 301012 or Placebo200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36 | Drug | Cohort C |
| ISIS 301012 or Placebo300 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, 36, 43, 50, 57, 64, 71, 78, and 85 | Drug | Cohort D |
Primary outcome
Percent reduction in LDL-cholesterol from baseline
Time frame: Week 7 (Cohorts A-C), Week 15 (Cohort D)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2016.
Related trials for Hypercholesterolemia, Familial
Active, not recruitingObservational study
Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
Familial Hypercholesterolaemia
Not yet recruiting
Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia
Not yet recruitingObservational study
An Observational Study to Evaluate Treatment Patterns in High, Very High and Extreme Cardiovascular Risk Patients With Hypercholesterolemia, Including Familial Hypercholesterolemia
Hypercholesterolaemia, Familial Hypercholesterolaemia
Active, not recruitingObservational study
Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia (HoFH), Major Adverse Cardiovascular Events (MACE)
Active, not recruitingObservational study
Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Genzyme, a Sanofi Company
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study of Hypercholesterolemia, Familial, run by Kastle Therapeutics, LLC at 8 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent reduction in LDL-cholesterol from baseline (time frame: Week 7 (Cohorts A-C), Week 15 (Cohort D)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Aug 2016.